2024 - Considerations for Oligonucleotide Therapeutics in CMC

Guidance in navigating through the regulatory grey area
Oligonucleotide therapeutics are an emerging field with over a dozen new oligonucleotide-based drugs approved globally since 2016. The well-established chemical process for synthesizing oligonucleotides gives access to ‘large small’ molecules of sizes between 5000 and 20,000 Daltons and positions oligonucleotide therapeutics between small molecules and biologicals.
Assessing the quality and specifications is often a challenge in this regulatory grey area because the International Council for Harmonisation (ICH) guidelines consider this modality as a chemical entity while sharing properties with biologics.
Therefore, a thorough understanding of oligonucleotide’s mechanism of action, safety aspects and the production process helps with informed decision-making for the chemistry, manufacturing and controls’ (CMC) critical quality attributes (CQAs).
In this webinar, the expert speakers will share insights on oligonucleotide therapeutics as an emerging therapeutic modality and focus on their production processes, drug substance specifications, material properties and potential impurities. They will also explore CMC Investigational Medicinal Product (IMP) considerations specific to oligonucleotides, including the identification of CQAs and critical process parameters (CPPs) essential for drug development.
They will also discuss key manufacturing aspects, as well as stability and scalability concerns for oligonucleotide therapeutics, to ensure a thorough understanding of the production lifecycle. Moreover, this webinar will provide guidance on progressing into early-phase clinical studies and emphasize on how to set and meet specifications to maintain quality and comply with regulatory standards, thus ensuring a smooth transition from development to clinical evaluation.
Register for this webinar today to gain expert insights into the complex regulatory landscape of oligonucleotide therapeutics.
Keywords: Small Molecule, Drug Development, Pharmaceutical Manufacturing, CRO, Drug Manufacturing, Regulatory, CMC, Commercial Manufacturing, Oligonucleotide Therapeutics